P757: INCIDENCE RATES OF HEALTHCARE RESOURCE UTILIZATION AND HEMOGLOBIN LEVELS DURING OPERA: A REAL-WORLD STUDY OF PEGCETACOPLAN TREATMENT IN US ADULTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
Authors: Jesse Fishman, Jinny Min, Lily Arnett, Apeksha Shenoy
Journal: HemaSphere (2023)

Topic: 11. Bone marrow failure syndromes incl. PNH - Biology & Translational Research Background: Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare (1-1.5 cases per million), acquired, life-threatening hematologic disease characterized by complement-mediated hemolysis and thrombosis. The chronic hemolysis observed in PNH can lead to anemia, which can result in dependance on transfusions. PNH symptoms such as chronic anemia can also lead to hospital and emergency room (ER) visits, increasing healthcare resource utilization (HCRU) for patients. Pegcetacoplan (PEG) is the first FDA approved C3 inhibitor for US adults with PNH, and the first to be EMA approved for the treatment of EU adults with PNH who are anemic after treatment with a C5 inhibitor for at least 3 months. Although multiple clinical trials have assessed the efficacy of PEG, there is limited information on the use of PEG in a real-world setting. Aims: We report preliminary trends in HCRU, in the form of transfusions, all-cause ER visits and hospitalizations, as well as hemoglobin (Hb) for patients in OPERA, a descriptive, observational, exploratory outcomes study, presenting real-world data on PEG treatment for US adults with PNH. Methods: This nationally representative study centrally recruited (via a pharmacy) patients ≥18 years of age, diagnosed with PNH and prescribed PEG by a licensed medical professional in the US. Patients consented then enrolled to participate in the study as approved by an institutional review board. OPERA collected information from routine care and did not direct any medical interventions. Patients provided baseline (BL) HCRU through online questionnaires while BL Hb was reported by the site (healthcare provider verified). Patients were monitored during monthly calls, reporting Hb and HCRU (subject to data availability) over a 1-year follow-up period. OPERA patients' PEG treatment initiation and pharmacy refill request timeline were analyzed to establish treatment compliance. Hemoglobin analysis only included patients who did not report a transfusion in the treatment period and reported both a BL and ≥1 follow-up Hb value. Given disease rarity, a small sample size was expected. Results: Over 12 months of OPERA, 43 patients with PNH enrolled in the study and completed at least 1 monthly call, with a mean age of 43.8 years and 53.5% were female. At BL, 10 (23.3%) patients were previously treated with eculizumab, 22 (51.2%) with ravulizumab, 8 (18.6%) with both, and 3 (7.0%) were not previously treated with a C5 inhibitor. At BL, 24 (55.8%) patients reported receiving transfusions prior to enrollment (Table), with 10 (23.3%) requiring ≥4 transfusions in the 12 months prior to enrolling in OPERA, and 21 (48.8%) reporting spending ≥3 hours at centers for transfusion care. Over the first 12 months of the study, after initiating PEG treatment, only 7 (16.3%) patients reported transfusions; the incidence rate per person year for OPERA patients was 0.8 (95% CI 0.6–1.0) for transfusions, 0.3 (95% CI 0.2–0.6) for ER visits and 0.2 (95% CI 0.1–0.5) for hospitalizations (Table). For 38 patients with available data, dosed at 1080 mg of PEG twice weekly or every 3 days, over 161.0 person months, the mean (range) PEG treatment compliance was 98.0% (87.0–100.0%) since OPERA enrollment. Of the 31 patients who met Hb analysis criteria, the mean (SD) BL Hb level was 8.7 (2.0) g/dL and the mean (SD) latest reported Hb level increased to 11.5 (1.8) g/dL, with a median (IQR) follow-up period of 6.0 (3.0) months. Summary/Conclusion: In this real-world study of US adults with PNH receiving PEG, patients experienced low incidence rates of associated HCRU and a positive trend in Hb levels, after initiating treatment.Keywords: Hemoglobin, Real world data, Paroxysmal nocturnal hemoglobinuria (PNH), Incidence